Market Access and Reimbursement Q&A with Vice President of Novartis, Tay Salimullah

Could you please introduce yourself?

My name is Tay Salimullah, and I am the Vice President, Global Head of Value and Access, at Novartis. In this role, my focus is on building a 21st century integrated access function to transform rare diseases through gene therapies.  I oversee translational access, gene therapy pricing and contracting, geneconomics, real-world data, and public policy. I have diverse global market access, pricing, and commercial experience across biopharmaceuticals, consultancy, social sector and private equity. Since joining Novartis in 2013, I have led strategic and operational teams across various divisions in Pharma, Region Europe, Oncology, Cell and Gene Therapies, and Group Global Health with a focus on inspiring associates to think early and differently about patient access and reimbursement. Before Novartis, I spent more than a decade with Pfizer in commercial, market access, and strategy development. In 2009, I completed a Global Health Fellowship, gaining in-field healthcare systems experience in Malawi. Lastly, I previously worked for a private investment group to incubate healthcare access across Asia. I have lived and worked in Europe, Asia, Africa and the US, and am passionate about breakthrough thinking to transform global patient access.

What are the main challenges facing market access to advanced therapies?

Generally, market access challenges vary based on country.

The value of most treatments is assessed sporadically over time because the reality is healthcare systems were mostly designed for ongoing chronic therapies.

We connect as frequently as possible with government bodies, local stakeholders, and more to discuss specific local access needs. We offer tailored solutions to countries to accelerate the opportunity to receive life-saving therapy to as many children as possible.

For example, our “Day One” access program in Europe – Children with spinal muscular atrophy (SMA) in its most common and severe form, SMA Type 1, generally experience progressive muscle weakness and paralysis shortly after birth. The window for intervention gets smaller with each passing day, and most children will not see the age of two if left untreated. So, when working with a disease like this, we had to design, and must maintain, a program which works within existing frameworks to open up patient options as early as possible in countries without pre-existing early access pathways. We did this by offering ministries of health and reimbursement bodies flexible options like retroactive rebates, deferred payments, outcomes-based rebates, and more. We’ll continue to search for, identify and address these challenges to fulfill our mission.

We are proud that, in working in close collaboration with local governments and healthcare systems, access pathways for Zolgensma are now in place in 28 countries.

How are you enjoying this Q&A? Let us know your thoughts, here >>

What are the main considerations for reimbursing an advanced therapy? And how early into development should financial plans be established?

We are proud to have created access pathways in more than 28 countries, and more than 2,000 patients have been treated worldwide with Zolgensma to date through clinical trials, managed access programs and in the commercial setting.

When working on reimbursement for an advanced therapy, it is imperative that the price reflects the transformative nature, benefit and long-term value of Zolgensma, but also takes into account income levels, local affordability barriers and economic realities to help improve the affordability of our medicines. Additionally, we strive to offer a variety of flexible and customizable options designed for cohorts or groups of eligible patients to enable access. These may include retroactive rebates, deferred payments, installment options, and outcomes-based rebates. We work in collaboration with partners across the industry – advocacy, government, key opinion leaders and more – to ensure these considerations are addressed. Zolgensma is consistently priced based on the value it provides to patients, caregivers and health systems; however, final pricing and reimbursement decisions are determined at the country level.

To continue to ensure gene therapies reach the patients who might benefit from them, it is critical to engage with stakeholders in a collaborative way. This means partnering with advocacy groups in the early stages of research and development to deeply understand the unmet needs with existing treatments, as well as working with local health authorities to understand the potential long-term value these products bring and to help tailor solutions to their local markets.

What are the main regulatory considerations for advanced therapies?

Gene therapies have transformed patient lives, with two therapies commercialized successfully in the United States to-date – one being Zolgensma. But that is just the beginning. Ultimately, scientific and technological advances will grow the number of therapies available and expand their application to impact more people and treat more diseases, including chronic disease.

We believe that health authorities globally will raise the bar earlier in development, to ensure delivery of safe and effective new gene therapies. For a time, there was an assumption that companies could file for approval of a gene therapy based on Phase 1, open-label studies. There will still be occasional approvals of this kind, but they will be rarer. While the threshold for approval of a gene therapy has always been high, the threshold for entry into clinical development now appears to be on the same level.

We have already seen increasing requirements across the industry from regulators that ask for both broader efficacy data to support use in different patient types, as well as more robust, long-term safety data, even in earlier phases of clinical study. This reflects the regulatory authorities’ increasing understanding of the field, and an increased focus on how a gene therapy program fits into the context of the broader treatment landscape.

Critical to note is the opportunity to combine earlier health technology thinking with regulatory endpoints to ensure payers will have the necessary endpoints to accelerate access upon health authority approval.

Additionally, we expect efficacy and safety will remain forefront in regulators’ minds and the focus will be increasingly tailored to specific therapies and landscapes. How the FDA evaluates data and approaches each program will be unique to the potential benefit/risk of that program. Companies developing candidates will need to look at the particulars of every patient population, therapy and disease combination, knowing it will be unique and that the application of safety and efficacy will be evaluated independently.

Lastly, gene therapies are unique in that they offer life-long benefits which cannot be effectively captured in time-limited clinical trials used to generate clinical evidence for traditional medicines. This creates opportunity for companies that can showcase robust efficacy data in areas of unmet need.

How important is it to build strong relationships between biotechs, manufacturers, regulators, and financiers? How can these relationships be built successfully?

It is critical to build relationships between biotechs, manufacturers, regulators and financiers. As the field expands to tackle not only rare diseases, but also more common conditions, partnership between stakeholders will be critical to build innovative and sustainable access solutions. At present we have a handful of approved advanced therapies, but we are on the cusp of progressing therapies for chronic conditions, making sustainable access solutions imperative moving forward.

What are the main differences and challenges between achieving market access in the US and Europe?

In the US, there is a largely centralized system for the approval and commercialization of the majority of products. Once a medicine has been approved by the Food and Drug Administration (FDA), it’s presumably adopted in medical practice and most – if not all – insurance companies will reimburse the product. Even with each state being governed by the same regulatory body, the reality is that our access teams must work payer by payer, plan by plan, to develop bespoke contracting solutions for coverage decisions.

Each European country has its own evaluation and access frameworks based on a wide range of clinical and economic criteria. So, once a product is approved by the European Medicines Agency (EMA), that does not give confirmation that it will be adopted into the individual markets, even if the EMA is their governing regulatory body. In most European countries, there are several more steps between EMA approval and patients receiving access to a transformative treatment. For instance, many countries have their own health technology assessment (HTA) bodies that decide whether an EMA-approved product is to be utilized, and another payer that decides whether to reimburse the product or not.

Currently, the value of treatments is evaluated over a temporary, recurring period. Chronic treatments are the norm for healthcare systems – in the US and Europe – a norm that one-time gene therapies do not conform to, and therefore do not fit within standard value assessment models. In the US, if a therapy’s value is recognized by the FDA, it is much more likely to be widely accepted into common practice. In Europe, on the other hand, there are many more challenges, as EMA approval doesn’t always translate to payer reimbursement or even country adoption. While this is true in many instances, we are grateful for our unprecedented partnerships with EU healthcare systems and payers to shape bespoke access pathways so patients can rapidly gain sustainable access.

The value of transformative therapies must be evaluated through the value it brings to patients and the healthcare system as a whole. Assessments should consider the total treatment cost over a lifetime as opposed to on a dose-by-dose basis, as due to the one-time nature of gene therapies, the overall costs of treatment can be reduced over a patient’s lifetime.

Early on, Novartis Gene Therapies started talks with government bodies and local stakeholders across Europe to ensure access to Zolgensma, offering flexible options such as retroactive rebates, deferred payment and instalment options, and outcomes-based rebates. It’s these tailored solutions that have enabled children with SMA and their families across Europe to experience the transformational benefit of gene therapy. So far, we’ve seen great success with our customized reimbursement solutions across Europe.

Why do you think Europe poses greater issues in achieving market access?

Health systems in general are not built to review and determine the value of one-time therapies, and current health technology appraisals and methods do not account for small patient populations, struggling to attribute value to non-health related benefits, such as caregiver quality of life.

Transformative treatments have exposed and tested the limitations of our value assessment and reimbursement systems. Access to treatment is still very variable across Europe, and with one-time gene therapies disrupting traditional reimbursement processes, early access solutions must be tailored to each market to grant timely access to treatment, ultimately resulting in improved clinical outcomes. However, our experience with Zolgensma is that existing frameworks and processes can be made fit for purpose if they are flexible.

Initiatives like our “Day One” access program are designed to work within existing pricing and reimbursement frameworks and recognize the nature of a one-time gene therapy for a devastating and progressive disease. To manage payor uncertainties, we are also implementing progressive outcomes based agreements to provide reimbursement to the approved label population.

Early on, Novartis Gene Therapies started talks with government bodies and local stakeholders across Europe to ensure access to Zolgensma, offering flexible options such as retroactive rebates, deferred payment and instalment options, and outcomes-based rebates. It’s these tailored solutions that have enabled children with SMA and their families across Europe to experience the transformational benefit of gene therapy. So far, we’ve seen great success with our customized reimbursement solutions across Europe.

As more breakthrough cell and gene therapies are discovered and continue to come to market, more customized commercial pathways will need to be established. These innovative pathways aren’t solely beneficial to patients, but have a knock-on effect on the healthcare systems, paving the way for future transformative treatments to reach patients as fast as possible. Transformative treatments have exposed and tested the limitations of our value assessment and reimbursement systems. However, our experience with Zolgensma is that existing frameworks and processes in Europe can be shaped to support sustainable access.

Find out more about and register your place at Phacilitate’s Advanced Therapies Europe 2022 here >>